Defeat Duchenne Canada is pleased to share the community letter provided by Wave Life Sciences. Wave thanks the Duchenne Community for participating in their clinical development program of WVE-N531, an investigational antisense oligonucleotide (ASO). ASOs are short, manufactured, single strands molecules that can alter RNA to reduce, restore, or modify a specific protein expression. The WVE-N531 molecule is designed to skip a mutation in exon 53, one of the exons that causes Duchenne muscular dystrophy (DMD).

In their current phase 2 trial, Wave has shared positive interim data that includes that WVE-N531:

• Was well tolerated with a good safety profile
• Got into the muscle tissue in high concentrations of ~41,000 ng/g
• Produced exon skipping of 57%
• Led to substantial dystrophin expression in ambulatory participants with DMD

Please see the community letter for more details about the phase 2 study and what happens next in the FORWARD-53 study.

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